Treatment for Amyloidosis

Treatment for Amyloidosis

AL primary amyloidosis treatment options

The primary goal of treating AL primary amyloidosis is to minimize amyloid deposition in the body's tissues/organs. While better treatment options are still vitally needed, there are many more options today than just a few years ago.

AL primary amyloidosis treatment team

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Typically, your treatment team will include:

AL primary amyloidosis medication therapy

Specific therapies often include treatment with a steroid and with chemotherapy drugs. The exact regimen depends on the specifics of your case, and balancing the beneficial effects of treatment with the potential side effects.

Bone Marrow Transplant

Chemotherapy and bone marrow transplant

Another mechanism to treat AL primary amyloidosis involves giving stronger chemotherapy to try to further eliminate the abnormal cells that produce the light chains. In this treatment approach, the chemotherapy is so strong that it depletes the body's own normal bone marrow cells.

Because of this, stem cells (collected from the patient before the chemotherapy is given) are given back to a patient after the chemotherapy, a procedure called a "bone marrow transplant" or a "stem cell transplant."

Risks of a bone marrow transplant in treating AL primary amyloidosis

While this had been the preferred treatment approach for AL primary amyloidosis for many years, a landmark study published in 2007 found that survival was generally better with conventional medical therapy (as described above) than with bone marrow transplants. This is likely due to the side effects and toxicities associated with treatment with the very strong chemotherapy which is administered during a bone marrow transplant.

For this reason, we recommend that most patients with AL primary amyloidosis undergo conventional therapy – though there are some cases (such as when disease progresses despite treatment with conventional therapy) in which bone marrow transplant represents the best option.

In addition, with a goal of helping both current and future patients with amyloidosis, the Stanford Amyloid Center also actively participates in and leads clinical trials. Your treatment team will tell you about any clinical trials which you are eligible for, and explain the potential benefits and risks of any trial to you.

ATTR amyloidosis treatment options

Familial ATTR amyloidosis

The key to success for patients with familial ATTR amyloidosis is early diagnosis. This is most effective if individuals who carry the mutation are identified by genetic screening when a family member is known to carry the disease. Genetic testing for familial ATTR amyloidosis is available through the Stanford Amyloid Center.

If an individual is identified as carrying the genetic mutation, he/she will be carefully screened for manifestations of the disease, and screening will continue on an annual or semi-annual basis. If familial ATTR amyloidosis is identified early on, progression can be halted with a liver transplantation, as the liver produces the abnormal transthyretin protein. Multiple investigational medications are being studied to see if they can slow or halt progression of familial ATTR amyloidosis.

Senile ATTR amyloidosis

Treatment is generally aimed at the symptoms of senile ATTR amyloidosis, such as the heart. Because the heart is the most commonly affected organ, your disease will be monitored and treated by a cardiologist.

New treatment modalities are constantly being developed and tested. We are currently enrolling patients affected by both forms of ATTR amyloidosis in a registry aimed at better understanding the natural course of the disease. Upcoming clinical trials may soon aim at inhibiting the transthyretin protein from forming into amyloid fibrils with a new medication.

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